What’s holding back cures? Our collective ignorance (and no, not a pharma conspiracy)

The birthers, it turns out, aren’t the only ones with wacky conspiracy theories; evidently a lot of people out there really think there are cures “They” don’t want you to know about.

In particular, there seems to be a surprisingly pervasive belief that drug companies aren’t working on cures for disease because it’s far more profitable to chronically maintain patients on medication.  This also explains (so the reasoning goes) why drug companies offer so many incremental therapeutics, and so few revolutionary treatments.

If only this explanation were true.

Drug companies, in fact, are desperate to identify radically improved treatments for dreadful disease.  Even if you don’t accept (as I’ve recently argued) that most industry researchers I know aspire to create such profoundly effective medicines, imagine the economic value of a drug that cured Parkinson’s Disease or pancreatic cancer – consider what such a therapy would be worth.

Exhibit A is the explosion of pharmaceutical interest in hepatitis C.  How do you explain the billions of dollars in investment– including an eye-popping $11.8B acquisition — if companies weren’t looking to cure patients?  The entire premise of this field has been coming up with approaches to cure more patients faster.

Of course, progress in HCV has been exceptional; by and large, medicine has seen mostly incremental gains, and has witnessed few transformative therapies.   Why?

The unfortunate truth is that drug companies really want to cure disease, but rarely know how.  Medical science simply isn’t up to the challenge.  Most diseases aren’t well enough understood to enable the rational development of truly transformative treatments.

When high-profile pharma studies fail – such as the slew of recent Phase 3 Alzheimer’s Disease trials – it’s fashionable to characterize them as yet another industry failure.  There’s some truth to this: the proximal cause may well be a poor decision to continue the development of a questionable drug.  But the root cause is likely insufficient understanding of disease pathophysiology.

We should also be careful about dismissing the value of incremental advances– a reflex I know I still have, although I’ve recognized the value of seemingly small tweaks from the time I was a resident.  Even today, when I critique (as derivative) formulation plays like liquid Ritalin, I’m glad to be reminded of the kids who stand to benefit from just such a medication.

 

What’s Next?

As the healthcare system looks more critically at value – demanding more evidence of effectiveness from providers and products alike – drug companies will be faced with two options.

The best choice, of course, would be to figure out how to come up with truly revolutionary treatments.  Perhaps unexpected insights will emerge from big data and the integration of phenotypic and genotypic information, in theframework of system biology; maybe a new therapeutic modality will arrive on the scene.  It’s possible intensified collaboration between academic and industry researchers will eventually yield something useful, or that open-data approaches (as championed by organizations like Sage Bionetworks[disclosure: I served as a founding advisor]) will achieve critical mass, and deliver impactful insights.  But unless something substantial changes, progress is likely to remain slow and stochastic, and truly game-changing novel therapeutics will continue to be the exceptions rather than the rule.

Given the ongoing challenges of creating transformative medications, there’s likely to be intensified focus on capturing, in a more granular fashion, the benefits of incrementally improved drugs; such assessments will not be a “nice to have” but a “must have,” table stakes for consideration by payors, and (to the extent these measures are used to demonstrate efficacy) regulators as well.  I also suspect pharmas will increasingly look to offer “solutions” (e.g. associated app or access to an online community) not just pills, to deliver value, though it’s unclear whether such approaches will either prove effective or represent an attractive value proportion for the relevant stakeholders.

In a sense, things would be much simpler if pharma really was as malevolent as some seem to fear; if only there was a secret refrigerator somewhere with cures to our deadliest diseases.  That would be a far more solvable problem than the one we actually must confront: science is extremely hard.

Even so, this doesn’t let pharma off the hook.  As savvy industry watchers such as Bernard Munos, David Grainger and others have pointed out (seehere as well), pharmas could run far more efficiently, and make better, smarter, and faster decisions.

But at the end of the day, the fundamental challenge in the creation of new medicines remains the complexity of the underlying science.  Improved operational efficiencies may help reduce the cost and time of failure, but new understanding – ideally coupled with improved empirical approaches permitting progress in the absence of such understanding — will be required for enduring success. 

 

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David
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